Category Archives: Gene Medicine

Streptococcus pneumoniae is an opportunistic pathogen that commonly infects young children and the elderly. This atlas will help researchers better understand how to treat these infections.

Streptococcus pneumoniae is an opportunistic pathogen that commonly infects young children and the elderly. This atlas will help researchers better understand how to treat these infections.The bacteria Streptococcus pneumoniae colonizes the nasopharynx and can cause pneumonia. Then, it can spread from the lungs to the bloodstream and cause organ damage. This opportunistic pathogen commonly infects young children, those who are immunocompromised and the elderly. In 2015, S. pneumoniae infections worldwide killed an estimated 192,000 to 366,000 children under age 5.

To understand how this pathogen adapts to different locations in the body, and also how the host responds to the invading microbe, researchers at the University of Alabama at Birmingham, the University of Maryland School of Medicine and Yale University School of Medicine measured bacterial and host gene expression at five different sites in a mouse model the nasopharynx, lungs, blood, heart and kidneys using three genetically different strains of S. pneumoniae.

Their resulting in vivo atlas of host-pathogen interactions at disease-relevant anatomical sites is now published in Proceeding of the National Academy of Sciences. The researchers identified shared and organ-specific transcriptomes of S. pneumoniae, and they showed that the bacterial and host gene expression profiles are highly distinct during asymptomatic colonization versus disease-causing infection.

This means the bacterium behaves differently, depending on which site it infects, and that the mouse organs, in turn, also respond differently to the presence of bacteria. Additionally, certain S. pneumoniae genes were found to always be highly expressed by all three strains of bacteria at all anatomical sites, which makes them ideal targets for new vaccines or therapies.

This was the first time that gene expression profiles during colonization and at multiple host infection sites were mapped from both the host and the pathogen perspectives.

We believe that the atlas of transcriptional responses during host-pathogen interactions presented here, the authors wrote, will constitute an essential resource for the pneumococcal and microbial pathogenesis research communities and serve as a foundation for identification and validation of key host and pneumococcal therapeutic targets in future studies.

Carlos J. Orihuela, Ph.D., professor in the UAB Department of Microbiology, and Herv Tettelin, Ph.D., professor in the University of Maryland School of Medicine Department of Microbiology and Immunology, are co-senior authors.

Carlos J. Orihuela, Ph.D.Besides a descriptive analysis of the transcriptomes, researchers confirmed their findings using bacterial mutants, in vivo challenge experiments and host treatments. In challenge experiments, the researchers found that an interferon beta anti-inflammatory treatment prevented the bacteria from invading vital organs and promoted host survival. This finding offers potentially new therapeutic avenues.

Symptoms of pneumococcal infection include fever, cough, shortness of breath, chest pain, stiff neck, confusion, increased sensitivity to light, joint pain, chills, ear pain, sleeplessness and irritability. While advances in antibiotics and the use of pneumococcal conjugate vaccines since 2000 have lowered deaths attributable to S. pneumoniae, the pathogen continues to show an increase in antibiotic resistance, and it also can switch to capsule types that are not covered by the current United States Food and Drug Administration-approved vaccines. Thus, pneumococcal infections continue to be a significant cause of illness and death.

Co-authors with Tettelin and Orihuela, in the study, An in vivo atlas of host-pathogen transcriptomes during Streptococcus pneumoniae colonization and disease, are Adonis DMello, Department of Microbiology and Immunology, University of Maryland School of Medicine; Ashleigh N. Riegler, Eriel Martnez, Sarah M. Beno and Tiffany Ricketts, UAB Department of Microbiology; and Ellen F. Foxman, Department of Laboratory Medicine, Yale University School of Medicine.

Support came from the Merck, Sharpe & Dohme Corp. Merck Investigator Studies Program award IISP ID#: 57329 and from National Institutes of Health grant AI114800.

Originally posted here:
An atlas of S. pneumoniae and host gene expression during colonization and disease - The Mix

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

Excerpt from:
Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

-Funding from the Bill & Melinda Gates Foundation will support research to enable CRISPR/Cas9-based therapies for HIV that can benefit patients worldwide-

ZUG, Switzerland and CAMBRIDGE, Mass., Dec. 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the receipt of a grant from the Bill & Melinda Gates Foundation to research in vivo gene editing therapies for the treatment of HIV.

While we have demonstrated the promise of CRISPR/Cas9 gene editing ex vivo in sickle cell disease and beta thalassemia, an in vivo approach to editing hematopoietic stem cells could allow the transformative benefit of CRISPR/Cas9 to reach a broader array of patients, including those in low resource settings that lack sufficient infrastructure for stem cell transplantation, said Tony Ho, M.D., Executive Vice President and Head of Research & Development at CRISPR Therapeutics. We look forward to working on new therapies that could contribute to the global effort to reduce the burden of HIV.

The grant builds upon CRISPR Therapeutics proprietary CRISPR/Cas9 gene editing technology and expertise in editing hematopoietic stem cells and contributes to efforts to accelerate transformative medicines for global health.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Ho in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the expected benefits of CRISPR Therapeutics research funded by the Bill & Melinda Gates Foundation and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be favorable and predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; potential impacts due to the coronavirus pandemic, such as the timing and progress of preclinical studies; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo are registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel EidesWCG on behalf of CRISPR+1-617-337-4167reides@wcgworld.com

Read more:
CRISPR Therapeutics Receives Grant to Advance In Vivo CRISPR/Cas9 Gene Editing Therapies for HIV - GlobeNewswire

Genetic Testing to Establish Strong Foothold in Current and Future Healthcare System

The notable rise in the demand for hereditary genetic testing over the past few years is one of the major factors that is expected to fuel the growth of the global genetic analysis services market in the upcoming decade. Technological advancements coupled with the drive to discover new and innovative genetic analysis techniques are set to shape the overall growth trajectory of the global genetic analysis services market during the forecast period. Over the past decade, the genome testing sector has witnessed consistent developments due to which, the global genetic analysis services market is anticipated to expand at an impressive rate during the assessment period.

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Hereditary genetic testing has emerged as ideal, and a rapidly evolving technology within the genetic analysis services market. This is likely to continue, owing to advancements in technology and findings of research activities. The increasing demand for improved and cutting-edge prediction and diagnostic tools and services coupled with surge in demand for disease monitoring is anticipated to play a key role in the overall growth of the global genetic analysis services market during the assessment period.

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Healthcare experts and credible researchers around the world are of the opinion that genetic testing is expected to be the future of the healthcare ecosystem. Advancements in the biomedical field coupled with the notable rise in the number of companies that are developing new genetic-testing kits are expected to augment the global genetic analysis services market during the forecast period. Moreover, as interest levels for precision medicine continues to witness sizeable growth around the world, as a result of which the demand for genetic analysis services is projected to grow at an impressive pace.

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Uptake of Next-generation Sequencing and Multi-gene Tests to Drive Market

Advancements in the genetic technology are likely to play an instrumental role in shaping the growth trajectory of the global genetic analysis services market during the forecast period. Furthermore, due to advancements in technology, the scope of genetic testing has widened by a considerable margin due to which, the demand for genetic analysis services is increasing. While genetic analysis services in the past were largely time-consuming and cumbersome, at present, increasing speed and availability of genomic testing are anticipated to present a plethora of opportunities to the players involved in the current market landscape for genetic analysis services.

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In addition, the gradual shift in the point of access to testing is evolving, as more number of consumers can avail genetic analysis services outside the healthcare setting. Advancements in genetic medicine at the back of advancements in technology are likely to bolster the growth of the global genetic analysis services market during the assessment period.

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Research and Development Activities in Full Swing amid COVID-19 Pandemic

Research and development activities are expected to continue in full swing amid the ongoing COVID-19 pandemic. The significant rise in the demand for genetic counseling services during the ongoing COVID-19 crisis is anticipated to generate consistent revenue for the players involved in the genetic analysis services market. Furthermore, researchers and scientists are increasingly focusing on discovering genetic mechanisms that are required to prevent the spread and transmission of the novel coronavirus disease. Genetic research is estimated to unlock various intricate details of the novel coronavirus, thereby opening up new opportunities for mitigation. The ongoing research pertaining to genetics and its correlation with the ongoing pandemic is expected to provide a detailed and microscopic understanding of the overall cellular mechanisms of the virus.

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Genetic Analysis Services Market: Uptake of Next-generation Sequencing and Multi-gene Tests to Drive Market - BioSpace

DUBLIN--(BUSINESS WIRE)--The "Regenerative Medicine in Pharma - Thematic Research" report has been added to ResearchAndMarkets.com's offering.

Regenerative medicine is a multidisciplinary field that seeks to develop the science and tools that can help repair, augment, replace, or regenerate damaged or diseased human cells, tissues, genes, organs, or metabolic processes, to restore normal function. It may involve the transplantation of stem cells, progenitor cells, or tissue, stimulation of the body's own repair mechanisms, or the use of cells as delivery vehicles for therapeutic agents such as genes and cytokines.

It is widely anticipated that Gene therapy is the most valuable regenerative medicine sector however, this market is also expected to be slowed down by high cost of therapies, which may limit its accessibility.

Existing programs will facilitate the approval and development of regenerative medicines, however, a reimbursement system especially for curative therapies is warranted.

The publisher's Regenerative Medicine in Pharma report combines primary research from a cross-specialty panel of experts with in-house analyst expertise to provide an assessment of the development landscape.

Scope

Reasons to Buy

Key Topics Covered:

For more information about this report visit https://www.researchandmarkets.com/r/dzfubj

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Thematic Research into the Global Regenerative Medicine in Pharma Market - Opportunities, Challenges, and Unmet Needs - ResearchAndMarkets.com -...

WASHINGTON, Dec. 15, 2020 /PRNewswire/ --Over the last 30 years, significant progress has been made in the fight against cancer. Researchers have expanded their understanding of how cancer develops and how to target medicines for specific cancer types. Since peaking in 1991, the death rate associated with cancer declined by 29%, which translates to 2.9 million fewer cancer deaths. The most recent data shows that between 2016 and 2017 alone, cancer death rates declined by 2.2%, the largest single-year drop ever recorded. Despite the challenges imposed by the COVID-19 pandemic, this momentum continues with biopharmaceutical companies focusing on research and development of innovative cancer therapies.

Still, cancer remains the second leading cause of death in the United States, accounting for 21% of all deaths. It is estimated that new cancer cases reached 1.8 million in 2020, increasing demand for earlier screening and diagnosis, as well as new treatments to address substantial unmet medical needs so patients can continue to live long and healthy lives.

To continue the progress and deliver hope to those battling cancer, biopharmaceutical research companies are working to develop more effective and better tolerated treatments.

A new report today from PhRMA finds that more than 1,300 medicines and vaccines for various cancers are currently in development, either in clinical trials or awaiting review by the U.S. Food and Drug Administration.

New medicines have played a key role in cancer survival gains, much of which are driven by advances in molecular and genomic research that have revealed the unique complexities of cancer and changed our understanding of the disease. Examples of the science behind potential new cancer treatments include:

The more than 1,300 medicines and vaccines in development represent an increased recognition among researchers that no two cancers are alike, which has led to further adoption of personalized medicine and the creation of treatments to target cancers specific to a single person. As researchers continue to explore life-saving methods and technologies to fight cancer, it is important we foster an innovation ecosystem that encourages ongoing research and development in this space.

To read the new report on medicines and vaccines in clinical testing for various cancers, click here.

Learn more about cancer at PhRMA.org/Cancer

SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)

http://phrma.org

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Report: More than 1,300 Medicines and Vaccines in Development to Help Fight Cancer - PRNewswire